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Long-term Safety and Efficacy of Efanesoctocog Alfa (BIVV001) in Previously Treated Patients With Hemophilia A (XTEND-ed)

Primary Purpose

Hemophilia A

Status
Active
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
efanesoctocog alfa (BIVV001)
Sponsored by
Bioverativ, a Sanofi company
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Hemophilia A

Eligibility Criteria

undefined - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion criteria :

For participants rolling over into Arm A

  • Participants who have completed the studies EFC16923, EFC16925, Arm B or Arm C of the current study, or any other potential BIVV001 study.
  • Male or Female For participants new to BIVV001 (Arm B and C)
  • Participants who have severe hemophilia A, defined as <1 IU/dL (<1%) endogenous FVIII activity as documented either by central laboratory testing at screening or in historical medical records from a clinical laboratory demonstrating <1% FVIII coagulant activity (FVIII:C) or a documented genotype known to produce severe hemophilia A.
  • Previous treatment for hemophilia A (prophylaxis or on-demand) with any recombinant and/or plasma-derived FVIII, or cryoprecipitate for at least 150 EDs or 50 EDs for participants aged <6 years.
  • Platelet count ≥100 000 cells/μL at screening.
  • A participant known to be human immunodeficiency virus (HIV) antibody positive, either previously documented or identified from screening assessments, must have the following results prior to enrollment: CD4 lymphocyte count >200 cells/mm³ and viral load of <400 000 copies/mL
  • Male
  • Only for Arm B: Chinese participants
  • Only for Arm C: planned major surgery within 6 months after Day 1.

Exclusion criteria:

For participants rolling over into Arm A

  • Positive inhibitor result, defined as ≥0.6 Bethesda units (BU)/mL.
  • Participation in another study. For participants new to BIVV001 (Arm B and Arm C)
  • Any concurrent clinically significant liver disease that, in the opinion of the Investigator, would make the participant unsuitable for enrollment. This may include, but is not limited to cirrhosis, portal hypertension, and acute hepatitis.
  • Serious active bacterial, fungal, or viral infection (other than chronic hepatitis or HIV) present within 30 days of screening.
  • Other known coagulation disorder(s) in addition to hemophilia A.
  • History of hypersensitivity or anaphylaxis associated with any FVIII product.
  • History of a positive inhibitor (to FVIII) test defined as ≥0.6 BU/mL, or any value greater than or equal to the lower sensitivity cut-off for laboratories with cut-offs for inhibitor detection between 0.7 and 1.0 BU/mL, or clinical signs or symptoms of decreased response to FVIII administrations. Family history of inhibitors will not exclude the participant.
  • Positive inhibitor test (FVIII) result, defined as ≥0.6 BU/mL at screening.
  • Treatment with acetylsalicylic acid (ASA) or antiplatelet agents that are not nonsteroidal anti-inflammatory drugs (NSAIDs) within 2 weeks prior to screening.
  • Treatment with NSAIDs greater than the maximum dose specified in the regional prescribing information within 2 weeks prior to screening.
  • Systemic treatment within 12 weeks prior to Screening with chemotherapy and/or other immunosuppressive drugs (except for the treatment of hepatitis C virus [HCV] or HIV).
  • Emicizumab use within the 20 weeks prior to screening.
  • Major surgery within 8 weeks prior to screening.

The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

Sites / Locations

  • Orthopaedic Institute for Children-Site Number:8400003
  • Children's Hospital Los Angeles-Site Number:8400009
  • University of California San Diego-Site Number:8400007
  • University of Florida Health-Site Number:8400008
  • Children's Healthcare of Atlanta-Site Number:8400016
  • Rush University Medical Center-Site Number:8400010
  • Children's Hospital Of Iowa-Site Number:8400011
  • University of Michigan Medical Center-Site Number:8400006
  • Michigan State University School Of Med-Site Number:8400002
  • Hemostasis and Thrombosis Center of Nevada-Site Number:8400001
  • New York Presbyterian Hospital/Weill Cornell Medical Center-Site Number:8400017
  • East Carolina University -2390 Hemby Ln-Site Number:8400015
  • Cincinnati Children's Hospital Medical Center-Site Number:8400012
  • Children's Research Institute-Site Number:8400013
  • Bloodworks Northwest-Site Number:8400005
  • Children's Hospital Of Wisconsin-Site Number:8400014
  • Investigational Site Number :0320001
  • Investigational Site Number :0320002
  • Investigational Site Number :0320003
  • Investigational Site Number :0360004
  • Investigational Site Number :0360001
  • Investigational Site Number :0360002
  • Investigational Site Number :0360003
  • Investigational Site Number :0560003
  • Investigational Site Number :0760001
  • Investigational Site Number :1000171
  • Investigational Site Number :1000172
  • Investigational Site Number :1240005
  • Investigational Site Number :1240004
  • Investigational Site Number :1240002
  • Investigational Site Number :1240001
  • Investigational Site Number :1560006
  • Investigational Site Number :1560002
  • Investigational Site Number :1560001
  • Investigational Site Number :1560003
  • Investigational Site Number :1560004
  • Investigational Site Number :1560005
  • Investigational Site Number :1560009
  • Investigational Site Number :1560010
  • Investigational Site Number :1560013
  • Investigational Site Number :1560007
  • Investigational Site Number :2500005
  • Investigational Site Number :2500004
  • Investigational Site Number :2500001
  • Investigational Site Number :2500003
  • Investigational Site Number :2500006
  • Investigational Site Number :2760304
  • Investigational Site Number :2760302
  • Investigational Site Number :2760001
  • Investigational Site Number :2760002
  • Investigational Site Number :3000001
  • Investigational Site Number :3480002
  • Investigational Site Number :3480004
  • Investigational Site Number :3480005
  • Investigational Site Number :3720001
  • Investigational Site Number :3800002
  • Investigational Site Number :3800001
  • Investigational Site Number :3800003
  • Investigational Site Number :3920425
  • Investigational Site Number :3920423
  • Investigational Site Number :3920426
  • Investigational Site Number :3920422
  • Investigational Site Number :3920421
  • Investigational Site Number :3920424
  • Investigational Site Number :4100603
  • Investigational Site Number :4100601
  • Investigational Site Number :4100600
  • Investigational Site Number :5280002
  • Investigational Site Number :5280001
  • Investigational Site Number :7240002
  • Investigational Site Number :7240001
  • Investigational Site Number :7520001
  • Investigational Site Number :7560001
  • Investigational Site Number :1580005
  • Investigational Site Number :1580001
  • Investigational Site Number :1580003
  • Investigational Site Number :1580002
  • Investigational Site Number :1580004
  • Investigational Site Number :7920004
  • Investigational Site Number :7920001
  • Investigational Site Number :7920003
  • Investigational Site Number :8260005
  • Investigational Site Number :8260001
  • Investigational Site Number :8260003
  • Investigational Site Number :8260004

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Experimental

Experimental

Arm Label

Arm A: Previously treated in BIVV001 study

Arm B: Newly initiated (China Only) in BIVV001

Arm C: Newly initiated in BIVV001 with planned major surgery

Arm Description

This arm includes all participants who have completed the previous phase 3 studies on BIVV001, as well as participants who have completed Arm B or Arm C of this study rolling over in Arm A, and participants who will have completed any future BIVV001 study who will be proposed to continue BIVV001 treatment. Participants in this arm will continue receiving BIVV001 prophylaxis treatment once-weekly (QW) for a total of 100 exposure days (EDs) cumulative from the parent study and this study. Participants will have the opportunity to continue in this study for up to 4 years, unless BIVV001 is commercially available in their applicable participating country.

This arm includes Chinese participants of any age who will be newly initiated on BIVV001 prophylaxis treatment once-weekly (QW) for 52 weeks. After 52 weeks of treatment in this arm B, participants will be able to roll into arm A.

This arm includes participants of any age who will be newly initiated on BIVV001 prophylaxis treatment once-weekly (QW) and will undergo planned major surgery after at least 6 initial EDs with BIVV001, and within 26 weeks from Day 1. After 52 weeks of treatment in arm C, participants will be able to roll into arm A.

Outcomes

Primary Outcome Measures

Number of participants with the occurrence of inhibitor development (neutralizing antibodies detected against factor VIII [FVIII])
The number of participants with the occurrence of inhibitor development (neuatralizing antibodies detected against factor VIII [FVIII]) as determined via the Nijmegen modified Bethesda assay.

Secondary Outcome Measures

Annual bleeding rate (ABR)
Annualized bleeding rate (ABR) for treated bleeding episodes and all bleeding episodes (including untreated bleeds).
Annualized bleeding rate (ABR) by type of bleed
Annualized bleeding rate (ABR) by type during prophylaxis treatment per study arm and parent study.
Annualized bleeding rate (ABR) by location
Annualized bleeding rate (ABR) by location during prophylaxis treatment per study arm and parent study.
Percentage of patients who maintain factor VIII (FVIII) above prespecified activity levels
Percentage of participants who maintain factor VIII (FVIII) activity levels over 7 days post dose during prophylaxis treatment per study arm and per parent study or arm.
Number of injections and dose of BIVV0001 to treat a bleeding episode
Percentage of bleeding episode treated with a single injection of BIVV001
Assessment of response to BIVV001 treatment of individual bleeding episodes
Assessment of response to BIVV001 treatment of individual bleeding episodes based on the International Society on Thrombosis and Haemostasis (ISTH) 4-point response scale
Physician's global assessment (PGA) of participants response to BIVV001
Physician's global assessment (PGA) of participant's response to BIVV001 treatment based on a 4-point response scale .
Total annualized BIVV001 consumption
Total annualized BIVV001 consumption per participant during prophylaxis treatment
Annualized joint bleeding rate (AJBR)
Target joint resolution
Target joint development, resolution and maintenance of target joint resolution based on ISTH criteria.
Change from baseline in Hemophilia Joint Health Score (HJHS)
Change from Baseline to the end of study visit in total score and domain scores (eg, swelling and strength) assessed by the Hemophilia Joint Health Score (HJHS)
Change from baseline in PROMIS-SF Physical Function
Change in Quality of Life (QoL) measures from baseline to end of study visit per study arm and per parent study arm: PROMIS-SF Physical Function (participants aged ≥18 years old).)
Change from baseline in Haem-A-QoL total score and physical health score
Change from baseline in Haemophilia QoL Questionnaire for Adults (Haem-A-QoL) total and physical health domain score on participants aged ≥17 years old.
Change from baselin in Haemo-QoL total score and physical health score
Change from baseline in Haemophilia QoL Questionnaire for Children (Haemo-QoL) total and physical health domain score on participants aged ≥4 to 16 years old and parent proxy for participants aged ≥4 to to <12 years old.
Number of participants with adverse events (AEs) and serious adverse events (SAEs)
Participants with occurrences of treatment emergent adverse events (AEs) and serious adverse events (SAEs).
Number of participants with the occurrence of embolic and thrombotic events
Participants with the occurrence of embolic and thrombotic events.
PK parameter: Maximum activity (Cmax)
PK parameter: Elimination half-life (t1/2)
PK parameter: Total clearance (CL)
PK parameter: Total clearance at steady state (CLss)
PK parameter: Accumulation index (AI)
PK parameter: Area under the activity time curve (AUC)
PK parameter: Volume of distribution at steady state (Vss)
PK parameter: Mean residence time (MRT)
PK parameter: Incremental recovery (IR)
PK parameter: Trough activity (Ctrough)
PK parameter: Time above FVIII activity levels
Investigators' or Surgeons' assessment of participant's hemostatic response to BIVV001 treatment
Investigators' or Surgeons' assessment of participant's hemostatic response to BIVV001 treatment on the ISTH 4 point response for surgical procedures scale.
Number of injections and dose to maintain hemostasis during perioperative period for major surgery
Total BIVV001 consumption during perioperative period for major surgery
Number and type of blood component transfusions used during perioperative period for major surgery
Estimated blood loss during perioperative period for major surgery

Full Information

First Posted
November 23, 2020
Last Updated
March 13, 2023
Sponsor
Bioverativ, a Sanofi company
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1. Study Identification

Unique Protocol Identification Number
NCT04644575
Brief Title
Long-term Safety and Efficacy of Efanesoctocog Alfa (BIVV001) in Previously Treated Patients With Hemophilia A
Acronym
XTEND-ed
Official Title
A Phase 3 Open-label, Multicenter Study of the Long-term Safety and Efficacy of Intravenous Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein (rFVIIIFc-VWF-XTEN; BIVV001) in Previously Treated Patients With Severe Hemophilia A
Study Type
Interventional

2. Study Status

Record Verification Date
March 13, 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
February 23, 2021 (Actual)
Primary Completion Date
February 12, 2027 (Anticipated)
Study Completion Date
February 12, 2027 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Bioverativ, a Sanofi company

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No

5. Study Description

Brief Summary
Primary Objective: - To evaluate the long-term safety of BIVV001 in previously treated subjects with hemophilia A Secondary Objectives: To evaluate the efficacy of BIVV001 as a prophylaxis treatment. To evaluate the efficacy of BIVV001 in the treatment of bleeding episodes. To evaluate BIVV001 consumption for prevention and treatment of bleeding episodes. To evaluate the effect of BIVV001 prophylaxis on joint health outcomes. To evaluate the effect of BIVV001 prophylaxis on Quality of Life (QoL) outcomes. To evaluate the safety and tolerability of BIVV001 treatment. To assess the PK of BIVV001 based on the one stage activated partial thromboplastin time (aPTT) and two-stage chromogenic FVIII activity assays (only applicable to Arm B). To evaluate the efficacy of BIVV001 for perioperative management
Detailed Description
Participants will receive BIVV001 once weekly for a total of at least 100 exposure days to BIVV001 (including exposure during a BIVV001 parent study, if applicable). Participants will have the opportunity to continue in this study for up to 4 years, unless BIVV001 is commercially available in their applicable participating country.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hemophilia A

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Model Description
It is a 3-arm study with single intervention
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
261 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Arm A: Previously treated in BIVV001 study
Arm Type
Experimental
Arm Description
This arm includes all participants who have completed the previous phase 3 studies on BIVV001, as well as participants who have completed Arm B or Arm C of this study rolling over in Arm A, and participants who will have completed any future BIVV001 study who will be proposed to continue BIVV001 treatment. Participants in this arm will continue receiving BIVV001 prophylaxis treatment once-weekly (QW) for a total of 100 exposure days (EDs) cumulative from the parent study and this study. Participants will have the opportunity to continue in this study for up to 4 years, unless BIVV001 is commercially available in their applicable participating country.
Arm Title
Arm B: Newly initiated (China Only) in BIVV001
Arm Type
Experimental
Arm Description
This arm includes Chinese participants of any age who will be newly initiated on BIVV001 prophylaxis treatment once-weekly (QW) for 52 weeks. After 52 weeks of treatment in this arm B, participants will be able to roll into arm A.
Arm Title
Arm C: Newly initiated in BIVV001 with planned major surgery
Arm Type
Experimental
Arm Description
This arm includes participants of any age who will be newly initiated on BIVV001 prophylaxis treatment once-weekly (QW) and will undergo planned major surgery after at least 6 initial EDs with BIVV001, and within 26 weeks from Day 1. After 52 weeks of treatment in arm C, participants will be able to roll into arm A.
Intervention Type
Drug
Intervention Name(s)
efanesoctocog alfa (BIVV001)
Intervention Description
Pharmaceutical form:Solution for Injection Route of administration: Intravenous
Primary Outcome Measure Information:
Title
Number of participants with the occurrence of inhibitor development (neutralizing antibodies detected against factor VIII [FVIII])
Description
The number of participants with the occurrence of inhibitor development (neuatralizing antibodies detected against factor VIII [FVIII]) as determined via the Nijmegen modified Bethesda assay.
Time Frame
Baseline to month 48
Secondary Outcome Measure Information:
Title
Annual bleeding rate (ABR)
Description
Annualized bleeding rate (ABR) for treated bleeding episodes and all bleeding episodes (including untreated bleeds).
Time Frame
Baseline to month 48
Title
Annualized bleeding rate (ABR) by type of bleed
Description
Annualized bleeding rate (ABR) by type during prophylaxis treatment per study arm and parent study.
Time Frame
Baseline to month 48
Title
Annualized bleeding rate (ABR) by location
Description
Annualized bleeding rate (ABR) by location during prophylaxis treatment per study arm and parent study.
Time Frame
Baseline to month 48
Title
Percentage of patients who maintain factor VIII (FVIII) above prespecified activity levels
Description
Percentage of participants who maintain factor VIII (FVIII) activity levels over 7 days post dose during prophylaxis treatment per study arm and per parent study or arm.
Time Frame
Baseline to month 48
Title
Number of injections and dose of BIVV0001 to treat a bleeding episode
Time Frame
Month 48
Title
Percentage of bleeding episode treated with a single injection of BIVV001
Time Frame
Month 48
Title
Assessment of response to BIVV001 treatment of individual bleeding episodes
Description
Assessment of response to BIVV001 treatment of individual bleeding episodes based on the International Society on Thrombosis and Haemostasis (ISTH) 4-point response scale
Time Frame
Baseline to month 48
Title
Physician's global assessment (PGA) of participants response to BIVV001
Description
Physician's global assessment (PGA) of participant's response to BIVV001 treatment based on a 4-point response scale .
Time Frame
Baseline to month 48
Title
Total annualized BIVV001 consumption
Description
Total annualized BIVV001 consumption per participant during prophylaxis treatment
Time Frame
Baseline to month 48
Title
Annualized joint bleeding rate (AJBR)
Time Frame
Baseline to month 48
Title
Target joint resolution
Description
Target joint development, resolution and maintenance of target joint resolution based on ISTH criteria.
Time Frame
Month 48
Title
Change from baseline in Hemophilia Joint Health Score (HJHS)
Description
Change from Baseline to the end of study visit in total score and domain scores (eg, swelling and strength) assessed by the Hemophilia Joint Health Score (HJHS)
Time Frame
Baseline to month 48
Title
Change from baseline in PROMIS-SF Physical Function
Description
Change in Quality of Life (QoL) measures from baseline to end of study visit per study arm and per parent study arm: PROMIS-SF Physical Function (participants aged ≥18 years old).)
Time Frame
Baseline to month 48
Title
Change from baseline in Haem-A-QoL total score and physical health score
Description
Change from baseline in Haemophilia QoL Questionnaire for Adults (Haem-A-QoL) total and physical health domain score on participants aged ≥17 years old.
Time Frame
Baseline to month 48
Title
Change from baselin in Haemo-QoL total score and physical health score
Description
Change from baseline in Haemophilia QoL Questionnaire for Children (Haemo-QoL) total and physical health domain score on participants aged ≥4 to 16 years old and parent proxy for participants aged ≥4 to to <12 years old.
Time Frame
Baseline to month 48
Title
Number of participants with adverse events (AEs) and serious adverse events (SAEs)
Description
Participants with occurrences of treatment emergent adverse events (AEs) and serious adverse events (SAEs).
Time Frame
Baseline to month 48
Title
Number of participants with the occurrence of embolic and thrombotic events
Description
Participants with the occurrence of embolic and thrombotic events.
Time Frame
Baseline to month 48
Title
PK parameter: Maximum activity (Cmax)
Time Frame
Baseline to week 52
Title
PK parameter: Elimination half-life (t1/2)
Time Frame
Baseline to week 26
Title
PK parameter: Total clearance (CL)
Time Frame
Baseline to week 26
Title
PK parameter: Total clearance at steady state (CLss)
Time Frame
Baseline to week 26
Title
PK parameter: Accumulation index (AI)
Time Frame
Baseline to week 26
Title
PK parameter: Area under the activity time curve (AUC)
Time Frame
Baseline to week 26
Title
PK parameter: Volume of distribution at steady state (Vss)
Time Frame
Baseline to week 26
Title
PK parameter: Mean residence time (MRT)
Time Frame
Baseline to week 26
Title
PK parameter: Incremental recovery (IR)
Time Frame
Baseline to week 52
Title
PK parameter: Trough activity (Ctrough)
Time Frame
Baseline to week 52
Title
PK parameter: Time above FVIII activity levels
Time Frame
Baseline to week 26
Title
Investigators' or Surgeons' assessment of participant's hemostatic response to BIVV001 treatment
Description
Investigators' or Surgeons' assessment of participant's hemostatic response to BIVV001 treatment on the ISTH 4 point response for surgical procedures scale.
Time Frame
Baseline to month 48
Title
Number of injections and dose to maintain hemostasis during perioperative period for major surgery
Time Frame
Baseline to month 48
Title
Total BIVV001 consumption during perioperative period for major surgery
Time Frame
Baseline to month 48
Title
Number and type of blood component transfusions used during perioperative period for major surgery
Time Frame
Baseline to month 48
Title
Estimated blood loss during perioperative period for major surgery
Time Frame
Baseline to month 48

10. Eligibility

Sex
All
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion criteria : For participants rolling over into Arm A Participants who have completed the studies EFC16923, EFC16925, Arm B or Arm C of the current study, or any other potential BIVV001 study. Male or Female For participants new to BIVV001 (Arm B and C) Participants who have severe hemophilia A, defined as <1 IU/dL (<1%) endogenous FVIII activity as documented either by central laboratory testing at screening or in historical medical records from a clinical laboratory demonstrating <1% FVIII coagulant activity (FVIII:C) or a documented genotype known to produce severe hemophilia A. Previous treatment for hemophilia A (prophylaxis or on-demand) with any recombinant and/or plasma-derived FVIII, or cryoprecipitate for at least 150 EDs or 50 EDs for participants aged <6 years. Platelet count ≥100 000 cells/μL at screening. A participant known to be human immunodeficiency virus (HIV) antibody positive, either previously documented or identified from screening assessments, must have the following results prior to enrollment: CD4 lymphocyte count >200 cells/mm³ and viral load of <400 000 copies/mL Male Only for Arm B: Chinese participants Only for Arm C: planned major surgery within 6 months after Day 1. Exclusion criteria: For participants rolling over into Arm A Positive inhibitor result, defined as ≥0.6 Bethesda units (BU)/mL. Participation in another study. For participants new to BIVV001 (Arm B and Arm C) Any concurrent clinically significant liver disease that, in the opinion of the Investigator, would make the participant unsuitable for enrollment. This may include, but is not limited to cirrhosis, portal hypertension, and acute hepatitis. Serious active bacterial, fungal, or viral infection (other than chronic hepatitis or HIV) present within 30 days of screening. Other known coagulation disorder(s) in addition to hemophilia A. History of hypersensitivity or anaphylaxis associated with any FVIII product. History of a positive inhibitor (to FVIII) test defined as ≥0.6 BU/mL, or any value greater than or equal to the lower sensitivity cut-off for laboratories with cut-offs for inhibitor detection between 0.7 and 1.0 BU/mL, or clinical signs or symptoms of decreased response to FVIII administrations. Family history of inhibitors will not exclude the participant. Positive inhibitor test (FVIII) result, defined as ≥0.6 BU/mL at screening. Treatment with acetylsalicylic acid (ASA) or antiplatelet agents that are not nonsteroidal anti-inflammatory drugs (NSAIDs) within 2 weeks prior to screening. Treatment with NSAIDs greater than the maximum dose specified in the regional prescribing information within 2 weeks prior to screening. Systemic treatment within 12 weeks prior to Screening with chemotherapy and/or other immunosuppressive drugs (except for the treatment of hepatitis C virus [HCV] or HIV). Emicizumab use within the 20 weeks prior to screening. Major surgery within 8 weeks prior to screening. The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Clinical Sciences & Operations
Organizational Affiliation
Sanofi
Official's Role
Study Director
Facility Information:
Facility Name
Orthopaedic Institute for Children-Site Number:8400003
City
Los Angeles
State/Province
California
ZIP/Postal Code
90007
Country
United States
Facility Name
Children's Hospital Los Angeles-Site Number:8400009
City
Los Angeles
State/Province
California
ZIP/Postal Code
90027
Country
United States
Facility Name
University of California San Diego-Site Number:8400007
City
San Diego
State/Province
California
ZIP/Postal Code
92121
Country
United States
Facility Name
University of Florida Health-Site Number:8400008
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32608
Country
United States
Facility Name
Children's Healthcare of Atlanta-Site Number:8400016
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Facility Name
Rush University Medical Center-Site Number:8400010
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60612-3833
Country
United States
Facility Name
Children's Hospital Of Iowa-Site Number:8400011
City
Iowa City
State/Province
Iowa
ZIP/Postal Code
52242
Country
United States
Facility Name
University of Michigan Medical Center-Site Number:8400006
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48109
Country
United States
Facility Name
Michigan State University School Of Med-Site Number:8400002
City
Lansing
State/Province
Michigan
ZIP/Postal Code
00000
Country
United States
Facility Name
Hemostasis and Thrombosis Center of Nevada-Site Number:8400001
City
Las Vegas
State/Province
Nevada
ZIP/Postal Code
89113
Country
United States
Facility Name
New York Presbyterian Hospital/Weill Cornell Medical Center-Site Number:8400017
City
New York
State/Province
New York
ZIP/Postal Code
10021
Country
United States
Facility Name
East Carolina University -2390 Hemby Ln-Site Number:8400015
City
Greenville
State/Province
North Carolina
ZIP/Postal Code
27834
Country
United States
Facility Name
Cincinnati Children's Hospital Medical Center-Site Number:8400012
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Facility Name
Children's Research Institute-Site Number:8400013
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43205-2696
Country
United States
Facility Name
Bloodworks Northwest-Site Number:8400005
City
Seattle
State/Province
Washington
ZIP/Postal Code
98104
Country
United States
Facility Name
Children's Hospital Of Wisconsin-Site Number:8400014
City
Milwaukee
State/Province
Wisconsin
ZIP/Postal Code
53226-0509
Country
United States
Facility Name
Investigational Site Number :0320001
City
Caba
State/Province
Ciudad De Buenos Aires
ZIP/Postal Code
C1425BWE
Country
Argentina
Facility Name
Investigational Site Number :0320002
City
Godoy Cruz
State/Province
Mendoza
ZIP/Postal Code
M5504FKD
Country
Argentina
Facility Name
Investigational Site Number :0320003
City
Buenos Aires
ZIP/Postal Code
C1015ABO
Country
Argentina
Facility Name
Investigational Site Number :0360004
City
Camperdown
State/Province
New South Wales
ZIP/Postal Code
2050
Country
Australia
Facility Name
Investigational Site Number :0360001
City
Westmead
State/Province
New South Wales
ZIP/Postal Code
2145
Country
Australia
Facility Name
Investigational Site Number :0360002
City
South Brisbane
State/Province
Queensland
ZIP/Postal Code
4101
Country
Australia
Facility Name
Investigational Site Number :0360003
City
Murdoch
State/Province
Western Australia
ZIP/Postal Code
6961
Country
Australia
Facility Name
Investigational Site Number :0560003
City
Bruxelles
ZIP/Postal Code
1200
Country
Belgium
Facility Name
Investigational Site Number :0760001
City
Campinas
State/Province
São Paulo
ZIP/Postal Code
13083-970
Country
Brazil
Facility Name
Investigational Site Number :1000171
City
Plovdiv
ZIP/Postal Code
4002
Country
Bulgaria
Facility Name
Investigational Site Number :1000172
City
Sofia
ZIP/Postal Code
1756
Country
Bulgaria
Facility Name
Investigational Site Number :1240005
City
Hamilton
State/Province
Ontario
ZIP/Postal Code
L8L 8E7
Country
Canada
Facility Name
Investigational Site Number :1240004
City
Hamilton
State/Province
Ontario
ZIP/Postal Code
L8N 3Z5
Country
Canada
Facility Name
Investigational Site Number :1240002
City
Ottawa
State/Province
Ontario
ZIP/Postal Code
K1H 8L1
Country
Canada
Facility Name
Investigational Site Number :1240001
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M5G 1X8
Country
Canada
Facility Name
Investigational Site Number :1560006
City
Beijing
ZIP/Postal Code
100006
Country
China
Facility Name
Investigational Site Number :1560002
City
Beijing
ZIP/Postal Code
100045
Country
China
Facility Name
Investigational Site Number :1560001
City
Guangzhou
ZIP/Postal Code
510515
Country
China
Facility Name
Investigational Site Number :1560003
City
Hangzhou
ZIP/Postal Code
310003
Country
China
Facility Name
Investigational Site Number :1560004
City
Hangzhou
ZIP/Postal Code
310003
Country
China
Facility Name
Investigational Site Number :1560005
City
Jinan
ZIP/Postal Code
250013
Country
China
Facility Name
Investigational Site Number :1560009
City
Kunming
ZIP/Postal Code
650032
Country
China
Facility Name
Investigational Site Number :1560010
City
Kunming
ZIP/Postal Code
650101
Country
China
Facility Name
Investigational Site Number :1560013
City
Lanzhou
ZIP/Postal Code
730000
Country
China
Facility Name
Investigational Site Number :1560007
City
Suzhou
ZIP/Postal Code
215006
Country
China
Facility Name
Investigational Site Number :2500005
City
Brest
ZIP/Postal Code
29200
Country
France
Facility Name
Investigational Site Number :2500004
City
Bron
ZIP/Postal Code
69500
Country
France
Facility Name
Investigational Site Number :2500001
City
Kremlin Bicetre
ZIP/Postal Code
94275
Country
France
Facility Name
Investigational Site Number :2500003
City
Lille
ZIP/Postal Code
59037
Country
France
Facility Name
Investigational Site Number :2500006
City
Marseille
ZIP/Postal Code
13385
Country
France
Facility Name
Investigational Site Number :2760304
City
Berlin
ZIP/Postal Code
10249
Country
Germany
Facility Name
Investigational Site Number :2760302
City
Bonn
ZIP/Postal Code
53127
Country
Germany
Facility Name
Investigational Site Number :2760001
City
Frankfurt am Main
ZIP/Postal Code
60590
Country
Germany
Facility Name
Investigational Site Number :2760002
City
München
ZIP/Postal Code
80337
Country
Germany
Facility Name
Investigational Site Number :3000001
City
Athens
ZIP/Postal Code
11527
Country
Greece
Facility Name
Investigational Site Number :3480002
City
Budapest
ZIP/Postal Code
1134
Country
Hungary
Facility Name
Investigational Site Number :3480004
City
Debrecen
ZIP/Postal Code
4093
Country
Hungary
Facility Name
Investigational Site Number :3480005
City
Pécs
ZIP/Postal Code
7623
Country
Hungary
Facility Name
Investigational Site Number :3720001
City
Dublin
ZIP/Postal Code
D12 N512
Country
Ireland
Facility Name
Investigational Site Number :3800002
City
Napoli
State/Province
Campania
ZIP/Postal Code
80123
Country
Italy
Facility Name
Investigational Site Number :3800001
City
Milano
ZIP/Postal Code
20121
Country
Italy
Facility Name
Investigational Site Number :3800003
City
Vicenza
ZIP/Postal Code
36100
Country
Italy
Facility Name
Investigational Site Number :3920425
City
Nagoya-shi
State/Province
Aichi
ZIP/Postal Code
466-0065
Country
Japan
Facility Name
Investigational Site Number :3920423
City
Kitakyushu-shi
State/Province
Fukuoka
ZIP/Postal Code
807-8555
Country
Japan
Facility Name
Investigational Site Number :3920426
City
Kawasaki-shi
State/Province
Kanagawa
ZIP/Postal Code
216-8511
Country
Japan
Facility Name
Investigational Site Number :3920422
City
Kashihara-Shi
State/Province
Niigata
ZIP/Postal Code
634-8521
Country
Japan
Facility Name
Investigational Site Number :3920421
City
Shinjuku-ku
State/Province
Tokyo
ZIP/Postal Code
160-0023
Country
Japan
Facility Name
Investigational Site Number :3920424
City
Suginami-ku
State/Province
Tokyo
ZIP/Postal Code
167-0035
Country
Japan
Facility Name
Investigational Site Number :4100603
City
Daegu
State/Province
Daegu-gwangyeoksi
ZIP/Postal Code
41404
Country
Korea, Republic of
Facility Name
Investigational Site Number :4100601
City
Seoul
State/Province
Seoul-teukbyeolsi
ZIP/Postal Code
03722
Country
Korea, Republic of
Facility Name
Investigational Site Number :4100600
City
Seoul
State/Province
Seoul-teukbyeolsi
ZIP/Postal Code
05278
Country
Korea, Republic of
Facility Name
Investigational Site Number :5280002
City
Amsterdam
ZIP/Postal Code
1105 AZ
Country
Netherlands
Facility Name
Investigational Site Number :5280001
City
Utrecht
ZIP/Postal Code
3584 CX
Country
Netherlands
Facility Name
Investigational Site Number :7240002
City
Esplugues de Llobregat
State/Province
Catalunya [Cataluña]
ZIP/Postal Code
08950
Country
Spain
Facility Name
Investigational Site Number :7240001
City
Madrid
State/Province
Madrid, Comunidad De
ZIP/Postal Code
28046
Country
Spain
Facility Name
Investigational Site Number :7520001
City
Malmö
ZIP/Postal Code
20502
Country
Sweden
Facility Name
Investigational Site Number :7560001
City
Zürich
ZIP/Postal Code
8032
Country
Switzerland
Facility Name
Investigational Site Number :1580005
City
Changhua County
ZIP/Postal Code
500
Country
Taiwan
Facility Name
Investigational Site Number :1580001
City
Taichung
ZIP/Postal Code
40201
Country
Taiwan
Facility Name
Investigational Site Number :1580003
City
Taichung
ZIP/Postal Code
407
Country
Taiwan
Facility Name
Investigational Site Number :1580002
City
Taipei
ZIP/Postal Code
10002
Country
Taiwan
Facility Name
Investigational Site Number :1580004
City
Taipei
ZIP/Postal Code
11031
Country
Taiwan
Facility Name
Investigational Site Number :7920004
City
Antalya
ZIP/Postal Code
07059
Country
Turkey
Facility Name
Investigational Site Number :7920001
City
Istanbul
ZIP/Postal Code
34390
Country
Turkey
Facility Name
Investigational Site Number :7920003
City
Izmir
ZIP/Postal Code
TR-35100
Country
Turkey
Facility Name
Investigational Site Number :8260005
City
London
State/Province
London, City Of
ZIP/Postal Code
NW3 2QG
Country
United Kingdom
Facility Name
Investigational Site Number :8260001
City
London
State/Province
London, City Of
ZIP/Postal Code
WC1N 3JH
Country
United Kingdom
Facility Name
Investigational Site Number :8260003
City
Birmingham
ZIP/Postal Code
B4 6NH
Country
United Kingdom
Facility Name
Investigational Site Number :8260004
City
Hampshire
ZIP/Postal Code
RG24 9NA
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing Plan Description
Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org

Learn more about this trial

Long-term Safety and Efficacy of Efanesoctocog Alfa (BIVV001) in Previously Treated Patients With Hemophilia A

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